Research Update: Discovery of Immune Cell's Molecular Signature Likely to Speed ALS Therapy Development

In work supported by The ALS Association, researchers have for the first time identified a unique molecular “signature” for a type of immune cell in the brain and spinal cord believed to contribute to ALS. The study was published in the journal Nature Neuroscience.

ALS (amyotrophic lateral sclerosis), often referred to as Lou Gehrig’s Disease, is a progressive neurodegenerative disease that affects neurons (nerve cells) in the brain and the spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. There is no cure and no life-prolonging treatments for the disease.

The research team investigated ways to distinguish cells called microglia, which reside in the brain and spinal cord, from other immune cells that migrate in during the ALS disease process. Under certain conditions, microglia are thought to accelerate ALS by contributing to “neuroinflammation,” an inflammatory process that contributes to the loss of motor neurons. Motor neurons are the major neuron type affected in ALS. They found that microglia could be identified by their expression of a unique set of genes and that this expression depended on a growth factor called TGF-beta.

“This should provide a basis for both understanding microglia biology and for modulating microglia in the treatment of central nervous system diseases,” said Oleg Butovsky, PhD, who led the study, along with Howard Weiner, MD. Dr. Butovsky is Instructor in Neurology at Harvard Medical School. Dr. Weiner is Professor of Neurology at Harvard.

“Microglia have emerged as potentially critical contributors to ALS,” said Lucie Bruijn, PhD, Chief Scientist for The Association, “and may be an important—and accessible—target for therapy to slow the progress of the disease. The more we can learn about their function, and how to precisely identify them, the faster we can translate that knowledge into new treatments.”

Dr. Butovsky is funded through a TREAT ALS™ Drug Discovery contract. The milestone-driven program enables the investigator to further develop a treatment approach and bring an important discovery closer to the clinic.

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